Adapting CRISPR/Cas9 System for Targeting Mitochondrial Genome

نویسندگان

چکیده

Gene editing of the mitochondrial genome using CRISPR-Cas9 system is highly challenging mainly due to sub-efficient delivery guide RNA and Cas9 enzyme complexes into mitochondria. In this study, we were able perform gene in DNA by appending an NADH-ubiquinone oxidoreductase chain 4 (ND4) targeting transport-derived stem loop element (RP-loop) expressing with a preceding localization sequence. We observe colocalization RP-loop gRNA marked reduction ND4 expression cells carrying 11205G variant their sequence coincidently decreasing mtDNA levels. This proof-of-concept study suggests that stem-loop added sgRNA can be transported mitochondria functionally interact mediate sequence-specific cleavage. Using novel approach target mtDNA, our results provide further evidence CRISPR-Cas9-mediated might potentially used treat mitochondrial-related diseases.

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ژورنال

عنوان ژورنال: Frontiers in Genetics

سال: 2021

ISSN: ['1664-8021']

DOI: https://doi.org/10.3389/fgene.2021.627050